Daybue becomes first Rett syndrome therapy to win approval in Canada
Treated patients in clinical trials showed gains in behavior, communication skills
Health Canada has approved Daybue (trofinetide) for the treatment of Rett syndrome in adults and children ages 2 and older who weigh at least 9 kg (about 19.8 lbs).
The decision makes Daybue the first approved Rett treatment in Canada, according to its developer Acadia Pharmaceuticals. The company said it will work to bring the newly approved treatment to patients as quickly as possible, but it didn’t give a specific timeline.
“Health Canada’s authorization of Daybue is a significant milestone for the Rett syndrome community in Canada and another step forward in Acadia’s commitment to increase access to this therapy for patients and their families,” Catherine Owen Adams, CEO of Acadia, said in a company press release. “We look forward to bringing Daybue to Canadian patients with Rett syndrome as soon as possible.”
Daybue aims to lessen inflammation, promote health of nerve cell connections
News of the approval was welcomed by the Ontario Rett Syndrome Association (ORSA), a Canadian advocacy organization.
“Today’s news is a historic milestone for the Canadian Rett syndrome community, who have eagerly awaited an approved treatment,” said Sabrina Millson, president of the ORSA. “Now that this therapy has been approved, we hope it will be made accessible through private and publicly funded drug plans as quickly as possible for all those who could benefit from it.”
Daybue was approved in the U.S. for Rett syndrome last year, and Acadia has announced plans to seek approvals in other markets, including Europe and Japan. The company’s application for approval in Canada was based primarily on data from the Phase 3 clinical trial LAVENDER (NCT04181723).
Results showed that Daybue was better than a placebo at normalizing measures of behavior and communication in girls and young women with Rett syndrome. Participants who completed LAVENDER could enter the extension studies LILAC-1 (NCT04279314) and LILAC-2 (NCT04776746), where Daybue was given to all for up to about three years. Results showed continual improvements in these two measures.
Rett is a genetic condition, with the disease’s symptoms marked by abnormalities in behavior and difficulties with communication. Daybue is a solution therapy taken daily by mouth or feeding tube to lessen inflammation, and to help create healthy connections between nerve cells to normalize brain activity. Common treatment side effects seen in its trials include diarrhea and vomiting.
“Rett syndrome is a debilitating and complex neurodevelopmental disorder that presents differently across patients and can lead to a range of symptoms throughout a patient’s life,” said Anita Datta, MD, co-director of the Rett Program at British Columbia Children’s Hospital. “Until now, treatment options have primarily focused on symptomatic management, as specific therapies for Rett syndrome have not existed.”
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